Stoke Therapeutics develops RNA medicines intended to restore protein expression, using its TANGO approach and antisense oligonucleotides for diseases caused by insufficient gene output. Its lead investigational medicine, zorevunersen, is in development for Dravet syndrome, with earlier work directed at rare genetic neurodevelopmental and other haploinsufficiency diseases.
Company news commonly covers zorevunersen clinical data, EMPEROR study updates, financial results, capital resources, collaboration activity, board and governance changes, equity inducement grants under Nasdaq rules, and healthcare conference presentations.
Stoke Therapeutics (STOK) appointed Bo Cumbo to its board on September 23, 2026, as director Edward Kaye retired. Kaye had served on the board since 2017 and was Stoke’s chief executive officer until March 2025. He will continue to advise the board through the transition. Cumbo has more than 30 years of biopharmaceutical experience and has been president and chief executive officer of Solid Biosciences since 2022.
Cumbo previously served as Sarepta Therapeutics’ chief commercial officer, where he worked on launches of treatments for Duchenne muscular dystrophy. Stoke is evaluating zorevunersen, an investigational treatment for Dravet syndrome, in a Phase 3 study. Stoke’s chief executive said the company is preparing for a potential FDA approval in early 2028. Zorevunersen’s safety and efficacy have not been evaluated by a regulatory authority. Stoke collaborates with Biogen (BIIB) on its development and commercialization.
Stoke Therapeutics (STOK) granted inducement stock options for an aggregate 53,175 common shares to nine new employees effective September 15, 2026, under Nasdaq Listing Rule 5635(c)(4).
The options have an exercise price of $28.15 per share, equal to the closing price of the common stock on the grant date, and a 10-year term. Each award vests over four years: 25% on the one-year anniversary of the applicable vesting commencement date, with the remaining 75% vesting in equal monthly instalments over the next 36 months, subject to continued employment and the terms of the 2023 Inducement Plan and the related option agreements. Stoke’s lead investigational medicine, zorevunersen, is in Phase 3 development for Dravet syndrome.
Stoke Therapeutics (STOK) and Biogen (BIIB) reported long-term clinical data for investigational Dravet syndrome therapy zorevunersen from Phase 1/2a and open-label extension studies, and detailed the ongoing global Phase 3 EMPEROR trial.
Four-year extension data showed durable reductions in seizures and statistically significant improvements in cognition and behavior at 1, 2, 3 and 4 years versus open-label baseline. Exploratory analyses indicated substantial reductions in the most severe seizure types through 3 years and improved quality of life through 28 months. Zorevunersen was generally well tolerated over more than 930 administered doses, with some patients treated for more than 5 years; elevated cerebrospinal fluid protein was common but without serious clinical manifestations or hydrocephalus. The EMPEROR study has completed enrollment in the United States, United Kingdom, Japan and Europe, with 162 patients in the primary analysis population and 34 in Europe, and a Phase 3 data readout is anticipated in Q3 2027 to complete a planned rolling U.S. NDA submission in the second half of 2027.
Stoke Therapeutics (STOK) will present at the Cantor Global Healthcare Conference on Wednesday, September 9, 2026, at 1:00 p.m. ET. CEO Ian F. Smith and Chief Patient Officer Jason Hoitt will speak, and a live webcast plus archived replay will be available in the Investors & News section of Stoke’s website.
Stoke Therapeutics (Nasdaq: STOK) and Biogen (Nasdaq: BIIB) are presenting new clinical data for zorevunersen, an investigational antisense oligonucleotide for Dravet syndrome, at the 16th European Epilepsy Congress in Athens, Sept. 5–9, 2026. The presentations include the first 4‑year results from ongoing Phase 1/2a open‑label extension (OLE) studies in highly refractory patients aged 2–18 years (N=81).
According to Stoke Therapeutics, 93% (75/81) of patients entered OLEs and 77% (58/75) remained on treatment at the 4‑year cutoff, with more than 930 doses given as of July 31, 2026. Zorevunersen showed sustained improvements in cognition and behavior, durable seizure reductions, and a generally well‑tolerated safety profile. Elevated cerebrospinal fluid (CSF) protein lab values occurred in about 94% of patients, with 59% classified as treatment‑emergent adverse events, but no serious or severe clinical manifestations or hydrocephalus were reported. Zorevunersen has orphan drug and Breakthrough Therapy designations and is being studied in the global pivotal Phase 3 EMPEROR trial, with results anticipated in the third quarter of 2027.
Stoke Therapeutics (Nasdaq: STOK) granted stock options to purchase an aggregate of 70,950 shares of common stock to seven new employees, effective August 17, 2026, as material inducement awards under Nasdaq Listing Rule 5635(c)(4) and the company’s 2023 Inducement Plan.
The options have a $32.13 exercise price, equal to the closing share price on August 17, 2026, a 10‑year term, and vest over four years: 25% after one year and the remainder monthly over 36 months, subject to continued employment. Stoke is a biotechnology company developing RNA medicines, including Phase 3 candidate zorevunersen for Dravet syndrome.
Stoke Therapeutics (Nasdaq: STOK) announced that CEO Ian F. Smith and Chief Patient Officer Jason Hoitt will present at the Canaccord Genuity 46th Annual Growth Conference on Wednesday, August 12, 2026 at 11:00 a.m. ET. A live webcast and archived replay will be available in the Investors & News section of Stoke’s website.
The company is developing its lead investigational antisense oligonucleotide, zorevunersen, a potential disease-modifying treatment for Dravet syndrome, currently in a Phase 3 study and partnered with Biogen (Nasdaq: BIIB) under a regional commercialization collaboration.
Stoke Therapeutics (Nasdaq: STOK) reported second quarter 2026 revenue of $9.3 million, down from $13.8 million a year earlier, and a net loss of $61.6 million, or $0.93 per share. For the first half of 2026, revenue was $15.6 million versus $172.4 million in 2025, with a net loss of $111.6 million compared to prior-year net income of $89.4 million.
As of June 30, 2026, Stoke held $354.3 million in cash, cash equivalents and marketable securities, supplemented by $65.7 million from a subsequent ATM sale, totaling about $420 million, which is expected to fund operations through a potential U.S. commercialization of zorevunersen in early 2028. Enrollment of 162 patients in the global Phase 3 EMPEROR study in Dravet syndrome is complete, with primary data expected in Q3 2027 to support a rolling U.S. NDA beginning Q1 2027. The Phase 1 OSPREY study of STK-002 in ADOA has completed dosing in the first cohort with no serious or severe safety events observed and a safety and efficacy readout anticipated in H1 2027.
Stoke Therapeutics (Nasdaq: STOK) will host a webcast and conference call on Monday, August 3, 2026, at 4:30 p.m. ET to provide second quarter 2026 business and financial updates. The event will stream via the Investors & News section of Stoke’s website, with research analysts registering separately for Q&A dial-in details and other participants joining a listen-only webcast. An archived replay will be accessible for at least 90 days. Stoke is developing the investigational antisense oligonucleotide zorevunersen for Dravet syndrome and has a strategic collaboration with Biogen (Nasdaq: BIIB) for its global commercialization rights.
Stoke Therapeutics (Nasdaq: STOK) granted inducement stock options under Nasdaq Listing Rule 5635(c)(4), effective July 15, 2026, to newly appointed Chief Scientific Officer Thomas McCauley and seven other new employees. McCauley received options to purchase 225,000 shares of common stock; the seven additional hires received options for an aggregate 83,070 shares.
According to Stoke Therapeutics, all options carry a $29.91 exercise price, equal to the July 15, 2026 closing price, have a 10-year term, and vest over four years (25% after one year, then monthly over 36 months), subject to continued employment and the 2023 Inducement Plan terms.